Nonsense mutation Duchenne muscular dystrophy (nmDMD) patients living in the European Union may soon experience relief from their condition due to the European Commission granting PTC Therapeutics, Inc. conditional marketing for Translarna™ (ataluren). PTC Therapeutics may now market Translarna in the 28 Member States of the European Union, as well as Iceland, Liechtenstein, and Norway.
“We are delighted that Translarna was approved for the treatment of nmDMD,” stated Stuart W. Peltz, PhD, CEO of PTC Therapeutics, in a news release from the company. “By targeting the underlying cause of DMD, it has the potential to change the course of the disease. We are moving rapidly to make this product available to patients in the European Union as we continue our global efforts to fulfill our vision of making Translarna available to all the boys it may benefit.”
Part of the conditional marketing authorization requires PTC Therapeutics to complete confirmatory Phase 3 trials evaluating Translarna in nmDMD patients and to submit additional safety and efficacy data from the trial. The trial, ACT DMD, is a 48-week randomized double-blind multicenter study of 174 nmDMD patients. Of primary interest is change in six-minute walk distance (6MWD). From baseline to Week 48, patients who received 40 mg/kg/day Translarna, given in three doses, saw only a 12.9 meter decline in 6MWD, while patients who received placebo saw a 44.1 meter decline–a significant difference of 31.3 meters. Secondary endpoints of stair climb and stair descend time-function tests showed similar results suggesting slowed nmDMD progression in Translarna-treated patients.
Severe adverse effects during the study were not connected to Translarna treatment and were infrequent. Nausea, vomiting, and headache were the most commonly reported adverse effects reported by patients using the recommended dose of Translarna. No patients chose to discontinue treatment due to adverse reactions.
Impressive results such as these give Translarna great potential for clinical impact. The Committee for Medicinal Products for Human Use of the European Medicines Agency stated that Translarna slows the loss of walking ability in nmDMD patients. “The world’s first approved treatment for the underlying cause of DMD marks a very important moment for patients and their families,” siad Mark Rothera, Chief Commercial Officer of PTC Therapeutics. “It is our highest priority to make Translarna available to patients and we will be working with regulators, payers, physicians, and patient organizations to make that a reality.”
Even the most severely affected patients may benefit from Translarna. In ACT DMD, patients who started with a 6MWD less than 350 meters had a 68-meter longer 6MWD when treated with Translarna rather than a placebo.
“We are grateful to the patients, families, advocacy groups, and physicians who have supported PTC Therapeutics through many years of research and development of Translarna,” said Dr. Peltz. “The DMD community has been waiting a long time for treatment options and this conditional approval marks and important day for us all.”